Duchenne Muscular Dystrophy
Conditions
Brief summary
To evaluate the long-term safety and tolerability of ACE-031 administration in subjects with Duchenne muscular dystrophy (DMD) who participated in Study A031-03. \[Note: This study was terminated based on preliminary safety data. Pending further analysis of safety data and discussion with health authorities, a new ACE-031 trial will be planned.\]
Interventions
BIOLOGICALACE-031 (Extension of cohort 1 from core study, A031-03)
ACE-031 0.5 mg/kg subcutaneously once every 4 weeks for 24 weeks.
BIOLOGICALACE-031 (Extension of cohort 2 from core study, A031-03)
Up to 1.0 mg/kg subcutaneously once every 2 weeks for 24 weeks.
BIOLOGICALACE-031 (Extension of cohort 3 from core study, A031-03)
Up to 2.5 mg/kg subcutaneously once every 4 weeks for 24 weeks.
Sponsors
Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
4 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Completion of participation in Study A031-03 and Investigator approval * Continuation of corticosteroid therapy at the same absolute dose and schedule as on Study A031-03
Exclusion criteria
* Participation in any other therapeutic clinical trial * Plans to have surgery during the course of the study
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of patients with adverse events. | From treatment initiation to End-of-Study Visit, approximately 24 weeks later. |
| Change in laboratory parameters and vital signs. | Baseline to End-of-Study Visit, approximately 24 weeks later. |
Secondary
| Measure | Time frame |
|---|---|
| Percent change in muscle strength score by hand-held myometry. | Baseline to End-of-Study Visit, approximately 24 weeks later. |
| Change in distance traveled in 6 minutes (standardized 6-Minute-Walk Test). | Baseline to End-of-Study Visit, approximately 24 weeks later. |
| Percent change in total lean body mass by DXA scan. | Baseline to End-of-Study Visit, approximately 24 weeks later. |
| Change in pulmonary function tests. | Baseline to End-of-Study Visit, approximately 24 weeks later. |
| Change in time to travel 10 meters (standardized 10-Meter-Walk/Run test). | Baseline to End-of-Study Visit, approximately 24 weeks later. |
| Percent change in total body and lumbar spine bone mineral density by DXA scan. | Baseline to End-of-Study Visit, approximately 24 weeks later. |
Countries
Canada
Outcome results
None listed